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Bioverativ scientists are committed to the discovery and development of new medicines. We conduct clinical trials to determine the safety and efficacy of investigational therapies and better understand how to use our approved products

Pursuing the discovery of safe, effective therapies

Clinical Trials

A key step in advancing a program from research into an approved medicine is conducting clinical trials to test the safety and efficacy of an investigational therapy. We also run clinical trials to better understand how to use our approved products. 

We are grateful to the members of our community that participate in the clinical trials for our potential therapies, including patients, health care professionals, hospitals, and clinics. Each person’s participation contributes tremendously to our understanding of disease and has the potential to benefit patients around the world.

 

 

Currently active clinical trials:

  • A Study to Assess the Efficacy and Safety of Sutimlimab (formerly BIVV009) in Participants With Primary Cold Agglutinin Disease Who Have a Recent History of Blood Transfusion

    Primary objective: To determine whether sutimlimab administration results in a greater than or equal to (>=) 2 gram per deciliter (g/dL) increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood transfusion during treatment in participants with primary cold agglutinin disease (CAD) who have a recent history of blood transfusion.

    Study ID: NCT03347396 “Cardinal”

    ​For US clinical trial information, visit CADStudies.com. For global clinical trial information, visit global.CADstudies.com.

  • A Study to Assess the Efficacy and Safety of Sutimlimab (formerly BIVV009) in Participants With Primary Cold Agglutinin Disease Without a Recent History of Blood Transfusion

    Primary objective: To determine whether sutimlimab administration results in a greater than or equal to (>=)1.5 gram per deciliter (g/dL) increase in hemoglobin (Hgb) level and avoidance of transfusion in participants with primary cold agglutinin disease (CAD) without a recent history of blood transfusion.

    Study ID: NCT03347422 “Cadenza”

    For US clinical trial information, visit CADStudies.com. For global clinical trial information, visit global.CADstudies.com.

  • A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment

    Primary objective: To describe the time to tolerization with rFVIIIFc in participants with hemophilia A with inhibitors undergoing ITI treatment for the first time.
    Study ID:  NCT03093480 “verITI-8”

  • Study of rFVIIIFc for ITI in Hemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies

    Primary objective: To describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc in patients with hemophilia A who have failed previous attempts at tolerization.
    Study ID: NCT03103542 “ReITIrate”

  • A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (BIVV001) in Previously Treated Adults With Severe Hemophilia A

    Primary objective: Assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients with severe hemophilia A.
    Study ID: NCT03205163 “EXTEN-A”

  • A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

    Primary objective: Evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.
    Study ID: NCT03653247

  • A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

    Primary objective: Evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia.
    Study ID: NCT03432364

  • An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A

    Primary objective: To evaluate the safety of rFVIIIFc  in previously untreated participants with severe hemophilia A
    Study ID: NCT02234323 “PUPS A-LONG”

  • Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B

    To evaluate the safety of rFIXFc in previously untreated participants with severe hemophilia B
    Study ID: NCT02234310 “PUPS B-LONG”

Beginning the process

Before a clinical trial on a potential therapy can start, Bioverativ submits an Investigational New Drug (IND) Application in the United States, or a Clinical Trial Application (CTA), as it’s called outside the U.S., to the local regulatory agency. We work with regulatory agencies around the world, including the Food and Drug Administration (FDA) in the U.S. and the European Medicines Agency (EMA) in Europe. The application describes the results of preclinical testing and clearly outlines the plan for conducting clinical trials.

01.

Phase I

Trials in Phase One assess safety and dosage, and are usually conducted with healthy volunteers. The goal is to learn how the potential therapy is absorbed, metabolized, and eliminated from the body, and to uncover its most frequent and serious side effects.

02.

Phase II

Trials in Phase Two gather preliminary data on how the potential therapy works for people with the target disease or condition. Some trials compare results for two different test groups: participants receiving the potential therapy and participants receiving a different treatment. Researchers continue to pay close attention to safety and possible side effects.

03.

Phase III

In this final phase, investigators gather more comprehensive information about a potential therapy’s safety and effectiveness. Trials might examine different patient populations, compare different dosage amounts of the potential therapy, or combine it with other treatments.

Once Phase Three is completed, the potential therapy can be filed with regulatory authorities like the FDA or EMA, which can then approve it as a therapy for public use.

04.

Post-market research

After a therapy has been approved, regulatory agencies often require additional studies. These post-marketing studies may evaluate the therapy on new patient populations, monitor the long-term impact or compare the therapy with other therapies.

05.